Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones

a technology of tocotrienol quinones and methylmalonic aciduria, which is applied in the direction of biocide, drug composition, metabolic disorder, etc., can solve the problems of kidney failure, kidney failure, pancreatitis, seizures, and possible death, and achieve seizures and death. coma, possible death, and the effect of reducing the risk of coma

Inactive Publication Date: 2015-02-26
BIOELECTRON TECH CORP
View PDF2 Cites 34 Cited by
  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

[0046]The present invention comprises multiple aspects, features and embodiments, where such multiple aspects, features and embodiments can be combined and permuted in any desired manner. These and other aspects, features and embodiments of the present invention will become evident upon

Problems solved by technology

MMA is accompanied by progressive encephalopathy, dehydration, feeding problems, developmental delays, failure to thrive, lethargy, recurrent yeast infections,

Method used

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones
  • Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones
  • Treatment of methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones

Examples

Experimental program
Comparison scheme
Effect test

example 1

MMA Cell Line Assay and Initial Screen for Effective Compound

[0113]Alpha-Tocotrienol quinone, its redox-silent version (the bis-Boc protected corresponding hydroquinone), and solvent controls were tested for their ability to rescue cells from MMA fibroblasts of the patient diagnosed with MMA, when the cells were stressed by addition of L-buthionine-(S,R)-sulfoximine (BSO), as described in Jauslin et al., Hum. Mol. Genet. 11(24):3055 (2002), Jauslin et al., FASEB J. 17:1972-4 (2003), and International Patent Application WO 2004 / 003565. EC50 concentrations of test compound and its redox-silent version were determined and compared. The following compound, 2-((6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethyl-bis(t-butyloxycarbonyl)benzene-1,4-diol, the bis-Boc protected hydroquinone form of alpha-tocotrienol quinone, was used as the “redox-silent” alpha tocotrienol quinone, or αTTQ-RS.

[0114]DMEM (Dulbecco's Modified Eagle Medium)—No Glucose, No pyruvate—G...

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to view more

PUM

PropertyMeasurementUnit
Percent by massaaaaaaaaaa
Percent by massaaaaaaaaaa
Massaaaaaaaaaa
Login to view more

Abstract

The present invention relates to methods of treating methylmalonic aciduria and other organic acidurias with tocotrienol quinones, including alpha-tocotrienol quinone, in order to alleviate symptoms of the disease.

Description

CROSS-REFERENCE TO RELATED APPLICATIONS[0001]This application claims priority benefit of U.S. provisional patent application No. 61 / 505,051, filed Jul. 6, 2011. The entire contents of that application are incorporated by reference herein.TECHNICAL FIELD OF THE INVENTION[0002]The present invention relates to a method of treating methylmalonic aciduria, isovaleric aciduria, and other organic acidurias with tocotrienol quinones (including tocotrienol hydroquinones), for example, alpha-tocotrienol quinone.BACKGROUND OF THE INVENTION[0003]Organic acidurias (also known as organic acidemias) are a set of disorders caused by defects in amino acid metabolism, resulting in an abnormal accumulation of acids in the body. See Rezvani I., “Defects in metabolism of amino acids,” in: Kliegman R M, Behrman R E, Jenson H B, Stanton B F, eds. Nelson Textbook of Pediatrics. 18th ed. Philadelphia, Pa.: Saunders Elsevier; 2007, Chap 85.[0004]Methylmalonic aciduria (methylmalonic acidemia, MMA) is caused ...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to view more

Application Information

Patent Timeline
no application Login to view more
IPC IPC(8): A61K31/122
CPCA61K31/122A61K31/05A61P3/00
Inventor MILLER, GUY M.DIONISI-VICI, CARLOBERTINI, ENRICOMARTINELLI, DIEGO
Owner BIOELECTRON TECH CORP
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Try Eureka
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap